Outcome of neurofibromatosis type 1 patients treated with first line vinblastine for optic pathway gliomas: A Canadian multicenter study.

2019 Background: To date, the first line chemotherapy treatment in the majority of countries for children with NF-1 and OPG is vincristine + carboplatin. Toxicity of this regimen consists mostly in neuropathy, allergic reactions, and hearing loss. Vinblastine has shown promising activity in a phase II study in children with recurrent/refractory low grade glioma (LGG). The aim of this study was to assess the activity of vinblastine in chemotherapy naive children, and to assess the toxicity profile. Methods: Patients < 18 years old with unresectable or progressive LGG were eligible if they had not received any previous treatment with chemotherapy or radiation. Vinblastine was administered weekly at a dose of 6 mg/m2 over a period of 70 weeks. Patients who showed progression on 2 consecutive imaging studies or evidence of clinical progression were removed from treatment. Results: Overall, the study enrolled 54 patients with LGG. A total of 13 patients (24.1%) had NF-1. Patients with NF-1were younger at diagn...